Problems arise when patients travel to places where regulation is not paramount and where treatment is questionable to begin with. A simple Internet search reveals that there are many companies willing to sell "stem cell therapies" for virtually any indication in the form of injections, pills and creams without proven scientific basis or medical merit. We have seen physicians claiming that any autologous procedure, one in which the patient's own tissue are used to isolate adult stem cells, are safe to use when adequately processed.
There is no telling, however, without any studies as to the purity of the cells being hawked by today's stem cell alchemists whose concoctions may contain useless cells, such as fat cells that could prove deadly if injected into the bloodstream, or worse, a population of malignant transformed cells that could be cancerous.
This is when we start to see morbidity and mortality associated with these treatments. Stem cell therapy is just the latest in a slew of modalities that will cause patients harm if we, as a nation, cannot bring standardization and regulation to an entire industry.
No responsible party will advocate that the FDA regulate the stem cell industry with less scrutiny and vigor than it does for other therapeutics. However, the FDA may become more responsive after a company has established defensible safety and efficacy profiles for its stem cell therapy.
Once a Phase I safety study has been inspected and the data has met the standards evaluating the chances of adverse events, the therapy should be launched into an accelerated approval process with abbreviated timelines, allowing our sickest patients swift access to safe medical technologies with extraordinary promise. The FDA could allow marketing approval for the treatment and allow remaining questions to be thoroughly answered after the market launch. This is not unlike the culture embodied in the oncology review division of the FDA, which has a culture of fast-tracking treatments that show tremendous results in trials to aid physicians treating our sickest cancer patients in highly controlled environments.
The seriousness of this situation cannot be understated. There are legitimate companies developing products that have real potential to create groundbreaking technology that can offer treatment for conditions that now have had little to no options. Bear in mind these are therapies that have been investigated and validated thoroughly in trials and retrials for close to two decades in multiple animal and human studies. It would be better to have a few years of well-supported products approved by the FDA that have not answered all the questions than several more years of charlatans conjuring stem cell therapies from their basements.
Steve Brozak is President of WBB Securities, an independent broker-dealer and investment bank specializing in biotechnology, medical devices and pharmaceutical research. Salman Punekar, MD is a medical consultant to WBB Securities. Emad Samad is a Managing Director of WBB Securities.